Predictive Factors of Mortality in Pediatric Pleuropulmonary Blastoma: A Surveillance Epidemiology and End Results Database Analysis

Abstract

ABSTRACT Purpose: Pleuropulmonary blastoma (PPB) is a rare malignancy associated with mutations in the DICER1 gene. Early-stage disease (PPB type I) mimics cystic lung malformations and develops in infants <1 year of age, and PPB type II and III arises in older children. The objective of this study was to analyze predictive factors of mortality in pediatric patients aged 0–19 years diagnosed with PPB between 2000 and 2019 in the USA. Methods: A retrospective analysis of pediatric patients (0–19 years) in the Surveillance Epidemiology and End Results database was conducted from 2000 to 2019 with a diagnosis of PPB using International Classification of Disease for Oncology, third edition code 8973/3 and rare tumor code 45. Demographics, incidence, staging, treatment, and mortality were extracted. A mortality risk predictive equation was developed using logistic regression. Statistical analysis was conducted through Microsoft Excel Analysis ToolPak and Solver. Results: There were a total of 71 new cases of PPB during the study period, with 16 (22%) deaths. The demographic analysis demonstrated that 40/71 (56.3%) patients were female, 57/71 (80.3%) were White, and 64/71 (90.1%) resided in metropolitan areas. Regression analysis demonstrated a statistically significant correlation between mortality and stage (P = 0.029), need for chemotherapy (P = 0.047), and female sex (P = 0.019). There was no significant correlation between mortality and need for radiation, race, or age at diagnosis. Multiple logistic regression analysis generated a predictive equation of mortality dependent on the stage of PPB, need for chemotherapy, and sex. This equation has an 82% accuracy, 81% sensitivity, and an 18% false positive rate. Conclusion: PPB is a rare disease. Distinguishing PPB from benign cystic lung malformations in infancy is important to avoid progression to Type II and III PPB. Advanced stages of PPB have a greater need for systemic chemotherapy and radiation with a poor prognosis.